Tammy McDaid, a mother from Swansea, is facing a financial battle to secure a life-saving treatment for her five-year-old son, Tate. Tate was diagnosed with Sanfilippo Syndrome, a rare and progressive neurodegenerative condition, when he was four years old. The disease, often described as childhood dementia, causes a gradual loss of physical and cognitive abilities.
High Cost of Gene Therapy
In September, the US Food and Drug Administration approved the first gene therapy for this condition. The treatment, known as FAYUVI, was developed by the pharmaceutical company Ultragenyx. While the approval offered hope to families worldwide, the price tag has created a significant barrier for those outside the United States.
The one-time treatment costs $3.95 million, which translates to approximately £2.95 million. Tammy McDaid stated that she spent months praying for the approval, only to be confronted by the reality of the cost. She contacted Ultragenyx to understand how international families without US health insurance could afford the procedure.
Ultragenyx responded that they cannot provide a single estimate for the total cost of care for international patients. The company noted that costs depend on individual medical needs and the treating institution. They recommended that families work with their healthcare providers to explore potential access pathways before assuming travel to the US is the only option.
Urgency for Tate McDaid
Sanfilippo Syndrome Type A, also known as Mucopolysaccharidosis type III, affects roughly 140 children in the UK. There is currently no cure for the condition. The new gene therapy is not a cure but can delay the onset of symptoms and help children remain active for longer.
The disease progresses more rapidly after the age of five. Tate is turning six this month, and his mother believes time is running out. She noted that Tate already shows signs of cognitive decline. The older he gets, the less effective the treatment may become, and the more abilities he will lose in the process.
Tate does not speak, but his mobility is central to his life. Tammy described him as the happiest little man, blissfully unaware of his condition. She is fighting to preserve his ability to move and interact with the world around him.
Health officials in Wales are currently in discussions with their US counterparts to determine if there are any potential access pathways for Tate. Staff from the Noah’s Ark Children’s Hospital in Cardiff are also discussing the situation with officials in the United States on the family’s behalf.
Tammy began raising money before the drug received FDA approval. She has so far collected more than £160,000, which covers only about five percent of the total estimated cost. She expressed hope that the pharmaceutical company might eventually offer a reduced rate, but she acknowledges that the sum required remains in the millions.
“If a millionaire turned around tomorrow and gave us the money, we’d go to the US straight away,” Tammy said. “Unfortunately, we’re in a world where we need millions, not hundreds of thousands.”
Source: BBC
